BACKGROUND: The management of biliary tract cancers (BTCs) is complex due to limited data on the optimal therapeutic approach. This phase II multicenter study evaluated the efficacy and tolerability of vandetanib monotherapy compared to vandetanib plus gemcitabine or gemcitabine plus placebo in patients with advanced BTC. PATIENTS AND METHODS: Patients were randomized in a 1:1:1 ratio to 3 treatment groups: vandetanib 300mg monotherapy (V), vandetanib 100mg plus gemcitabine (V/G), gemcitabine plus placebo (G/P). Vandetanib (300mg or 100mg) or placebo were given in single oral daily doses. Gemcitabine 1000mg/m2 was intravenously infused on day 1 and day 8 of each 21-day cycle. The primary endpoint was progression-free survival (PFS). Secondary endpoints were: objective response rate (ORR), disease control rate, overall survival, duration of response, performance status and safety outcomes. RESULTS: 173 patients (mean age 63.6 years) were recruited at 19 centers across Italy. Median (95% confidence intervals) PFS (days) were 105 (72-155), 114 (91-193) and 148 (71-225) respectively for the V, V/G and G/P-treatment groups, with no statistical difference among them (P=0.18). No statistical difference between treatments was observed for secondary endpoints, except ORR, which slightly favored the V/G combination over other treatments. The proportion of patients reporting adverse events (AEs) was similar for the 3 groups (96.6% in V arm, 91.4% in the V/G arm and 89.3% in the G/P arm). CONCLUSIONS: Vandetanib treatment did not improve PFS in patients with advanced BTC. The safety profile of vandetanib did not show any additional AEs or worsening of already known AEs. CLINICAL TRIAL NUMBER: NCT00753675.
A randomized, multicenter, phase II study of vandetanib monotherapy versus vandetanib in combination with gemcitabine versus gemcitabine plus placebo in subjects with advanced biliary tract cancer: the VanGogh study.
Gebbia VInvestigation
;
2015-01-01
Abstract
BACKGROUND: The management of biliary tract cancers (BTCs) is complex due to limited data on the optimal therapeutic approach. This phase II multicenter study evaluated the efficacy and tolerability of vandetanib monotherapy compared to vandetanib plus gemcitabine or gemcitabine plus placebo in patients with advanced BTC. PATIENTS AND METHODS: Patients were randomized in a 1:1:1 ratio to 3 treatment groups: vandetanib 300mg monotherapy (V), vandetanib 100mg plus gemcitabine (V/G), gemcitabine plus placebo (G/P). Vandetanib (300mg or 100mg) or placebo were given in single oral daily doses. Gemcitabine 1000mg/m2 was intravenously infused on day 1 and day 8 of each 21-day cycle. The primary endpoint was progression-free survival (PFS). Secondary endpoints were: objective response rate (ORR), disease control rate, overall survival, duration of response, performance status and safety outcomes. RESULTS: 173 patients (mean age 63.6 years) were recruited at 19 centers across Italy. Median (95% confidence intervals) PFS (days) were 105 (72-155), 114 (91-193) and 148 (71-225) respectively for the V, V/G and G/P-treatment groups, with no statistical difference among them (P=0.18). No statistical difference between treatments was observed for secondary endpoints, except ORR, which slightly favored the V/G combination over other treatments. The proportion of patients reporting adverse events (AEs) was similar for the 3 groups (96.6% in V arm, 91.4% in the V/G arm and 89.3% in the G/P arm). CONCLUSIONS: Vandetanib treatment did not improve PFS in patients with advanced BTC. The safety profile of vandetanib did not show any additional AEs or worsening of already known AEs. CLINICAL TRIAL NUMBER: NCT00753675.I documenti in IRIS sono protetti da copyright e tutti i diritti sono riservati, salvo diversa indicazione.
